Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants

NCT05813314 | Terminated Phase 1 FDA Drug FDA Device

• 1 other identifier: 111-104
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to establish the bioequivalence of a single SC dose of BMN 111 administered using a vial and syringe versus using an injector pen.

Conditions

Achondroplasia

Outcome Measures

Primary Outcomes (3)

• Maximum observed drug concentration (Cmax)

  Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose

• Area under the concentration versus time curve from time 0 to infinity (AUC0-∞)

  Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose

• Area under the concentration versus time curve from time 0 to t, where t is the last timepoint with a measurable concentration (AUC0-t)

  Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose

Secondary Outcomes (4)

• Time of Cmax (tmax)

  Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose

• Time of t1/2

  Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose

• Time of terminal rate constant (λZ)

  Predose, 2, 5, 15, 30, 45, 60, and 75 minutes and 1.5, 2, 2.5, 3, 4, 6, and 8 hours postdose

• Incidence and severity of adverse events

  2 weeks

Study Arms (2)

BMN 111 injection with vial and syringe

ACTIVE COMPARATOR

Study treatment will be provided in glass vials. Each glass vial will be labeled as required per country requirement. Pre-filled Diluent Transfer Syringes will be provided for reconstitution.

Drug: BMN 111 administration via vial and syringe

BMN 111 injection with injector pen

EXPERIMENTAL

Study treatment will be provided in a prefilled injector pen containing a dual chamber drug cartridge, for reconstitution and injection, after setting of the specified dose with the 2.0 mg/mL formulation.

Combination Product: BMN 111 administration via Injector Pen

Interventions

BMN 111 administration via Injector Pen COMBINATION_PRODUCT

BMN 111 Injection will be given using an injector pen

BMN 111 injection with injector pen

BMN 111 administration via vial and syringe DRUG

BMN 111 Injection will be given using vial and syringe

BMN 111 injection with vial and syringe

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Participants must be aged 18 to 55 years inclusive, at the time of signing the informed consent.
• Participants must be non-smoking (have never smoked, or stopped smoking \> 3 months prior to dosing), and can be of any ethnicity.
• Participants must be healthy as determined by the absence of clinically relevant abnormalities identified by medical evaluation, including medical history, full physical examination, laboratory tests, cardiac monitoring, supine vital signs to include temperature, and assessment of prior and concomitant medications.
• Body weight ≥ 60 kg and \< 110 kg and body mass index within the range 18.0 to 32.0 kg/m2 (inclusive).
• Males and/or females are eligible to participate in this clinical study.
• Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
• Females will not be pregnant or lactating, and females of childbearing potential and males will agree to use contraception.
• Participants must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF).

You may not qualify if:

• Any condition considered to affect drug absorption (eg, conditions affecting SC administration such as reduced SC blood flow, lipohypertrophy, and significantly high/low skin temperature).
• Significant history or clinical manifestation of any metabolic, allergic, dermatological, hepatic, renal, hematological, pulmonary, cardiovascular, gastrointestinal, neurological, respiratory, endocrine, or psychiatric disorder, as determined by the investigator (or designee).
• History of significant hypersensitivity, intolerance, or allergy to any drug compound, food, or other substance, unless approved by the investigator (or designee).
• History of migraines.
• Confirmed (eg, two consecutive measurements) systolic blood pressure \> 140 or \< 90 mmHg, diastolic blood pressure \> 90 or \< 50 mmHg, and pulse rate \> 100 or \< 40 beats per minute or recurrent symptomatic hypotension (defined as episodes of low BP generally accompanied by symptoms e.g., dizziness and fainting) or recurrent symptomatic orthostatic hypotension.
• Positive hepatitis panel and/or positive human immunodeficiency virus test.
• Exposure to mycobacterium tuberculosis, indicative of tuberculosis infection, as confirmed by a positive QuantiFERON-TB Gold test.
• A positive COVID-19 test at Screening or Check-in.
• Taking any of the prohibited medications.
• Administration of a coronavirus disease 2019 (COVID-19) vaccine within 30 days prior to dosing.
• Use or intend to use any medications/products known to alter drug absorption, metabolism, or elimination processes, including St. John's wort, within 30 days prior to dosing, unless deemed acceptable by the investigator (or designee).
• Use or intend to use any prescription medications/products other than hormone replacement therapy (HRT), oral, implantable, transdermal, injectable, or intrauterine contraceptives within 7 days (or 14 days if the drug is a potential enzyme inducer), or 5 half-lives (whichever is longer), prior to dosing, unless deemed acceptable by the investigator (or designee).
• Use or intend to use slow release medications/products considered to still be active within 14 days prior to Check-in, unless deemed acceptable by the investigator (or designee).
• Use or intend to use any nonprescription medications/products including vitamins, minerals, and phytotherapeutic/herbal/plant derived preparations within 7 days, or 5 half-lives (whichever is longer), prior to dosing, unless deemed acceptable by the investigator (or designee).
• Participation in a clinical study involving administration of an investigational drug (new chemical entity) in the past 30 days or 5 half-lives prior to dosing (whichever is longer).

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (1)

Labcorp

Dallas, Texas, 75230, United States

Location

MeSH Terms

Conditions

Achondroplasia

Interventions

Syringes

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Osteochondrodysplasias; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Equipment and Supplies

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 16, 2023
• First Posted: April 14, 2023
• Study Start: March 15, 2023
• Primary Completion: June 14, 2023
• Study Completion: June 14, 2023
• Last Updated: October 12, 2023

Record last verified: 2023-09

Locations

US (1)